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Page 1
Patient-Customized Oligonucleotide Therapy for a Rare Genetic Disease.
Kim J, Hu C, Moufawad El Achkar C, Black LE, Douville J, Larson A, Pendergast MK, Goldkind SF, Lee EA, Kuniholm A, Soucy A, Vaze J, Belur NR, Fredriksen K, Stojkovska I, Tsytsykova A, Armant M, DiDonato RL, Choi J, Cornelissen L, Pereira LM, Augustine EF, Genetti CA, Dies K, Barton B, Williams L, Goodlett BD, Riley BL, Pasternak A, Berry ER, Pflock KA, Chu S, Reed C, Tyndall K, Agrawal PB, Beggs AH, Grant PE, Urion DK, Snyder RO, Waisbren SE, Poduri A, Park PJ, Patterson A, Biffi A, Mazzulli JR, Bodamer O, Berde CB, Yu TW. Kim J, et al. Among authors: goodlett bd. N Engl J Med. 2019 Oct 24;381(17):1644-1652. doi: 10.1056/NEJMoa1813279. Epub 2019 Oct 9. N Engl J Med. 2019. PMID: 31597037 Free PMC article.
Targeting neurological abnormalities in lysosomal storage diseases.
van Gool R, Tucker-Bartley A, Yang E, Todd N, Guenther F, Goodlett B, Al-Hertani W, Bodamer OA, Upadhyay J. van Gool R, et al. Trends Pharmacol Sci. 2022 Jun;43(6):495-509. doi: 10.1016/j.tips.2021.11.005. Epub 2021 Nov 27. Trends Pharmacol Sci. 2022. PMID: 34844772 Review.
A multidisciplinary assessment of pain in juvenile idiopathic arthritis.
Upadhyay J, Lemme J, Cay M, Van Der Heijden H, Sibai D, Goodlett B, Lo J, Hoyt K, Taylor M, Hazen MM, Halyabar O, Meidan E, Schreiber R, Chang MH, Nigrovic PA, Jaimes C, Henderson LA, Ecklund K, Sundel RP. Upadhyay J, et al. Semin Arthritis Rheum. 2021 Aug;51(4):700-711. doi: 10.1016/j.semarthrit.2021.05.011. Epub 2021 Jun 4. Semin Arthritis Rheum. 2021. PMID: 34139523 Free PMC article.
Proteomics based markers of clinical pain severity in juvenile idiopathic arthritis.
Van Der Heijden H, Fatou B, Sibai D, Hoyt K, Taylor M, Cheung K, Lemme J, Cay M, Goodlett B, Lo J, Hazen MM, Halyabar O, Meidan E, Schreiber R, Jaimes C, Ecklund K, Henderson LA, Chang MH, Nigrovic PA, Sundel RP, Steen H, Upadhyay J. Van Der Heijden H, et al. Pediatr Rheumatol Online J. 2022 Jan 15;20(1):3. doi: 10.1186/s12969-022-00662-1. Pediatr Rheumatol Online J. 2022. PMID: 35033099 Free PMC article.
First 1.5 years of pegvaliase clinic: Experiences and outcomes.
Sacharow S, Papaleo C, Almeida K, Goodlett B, Kritzer A, Levy H, Martell L, Wessel A, Viau K. Sacharow S, et al. Mol Genet Metab Rep. 2020 May 25;24:100603. doi: 10.1016/j.ymgmr.2020.100603. eCollection 2020 Sep. Mol Genet Metab Rep. 2020. PMID: 32489881 Free PMC article.
Safety and pharmacodynamics of an engineered E. coli Nissle for the treatment of phenylketonuria: a first-in-human phase 1/2a study.
Puurunen MK, Vockley J, Searle SL, Sacharow SJ, Phillips JA 3rd, Denney WS, Goodlett BD, Wagner DA, Blankstein L, Castillo MJ, Charbonneau MR, Isabella VM, Sethuraman VV, Riese RJ, Kurtz CB, Brennan AM. Puurunen MK, et al. Among authors: goodlett bd. Nat Metab. 2021 Aug;3(8):1125-1132. doi: 10.1038/s42255-021-00430-7. Epub 2021 Jul 22. Nat Metab. 2021. PMID: 34294923 Clinical Trial.
Publisher Correction: Safety and pharmacodynamics of an engineered E. coli Nissle for the treatment of phenylketonuria: a first-in-human phase 1/2a study.
Puurunen MK, Vockley J, Searle SL, Sacharow SJ, Phillips JA 3rd, Denney WS, Goodlett BD, Wagner DA, Blankstein L, Castillo MJ, Charbonneau MR, Isabella VM, Sethuraman VV, Riese RJ, Kurtz CB, Brennan AM. Puurunen MK, et al. Among authors: goodlett bd. Nat Metab. 2022 Sep;4(9):1214. doi: 10.1038/s42255-022-00635-4. Nat Metab. 2022. PMID: 35974248 No abstract available.
14 results